RNA interference therapeutics for cancer: Challenges and opportunities (Review)

作者: Roop Bora

DOI: 10.3892/MMR.2012.871

关键词: GeneSmall interfering RNAGene silencingRNA silencingRNACancerRNA interferenceBioinformaticsBiologySmall molecule

摘要: RNA interference (RNAi) is a sequence-specific, post-transcriptional gene silencing mechanism in animals and plants, which mediated by double-stranded (dsRNA). There has recently been an increasing interest harnessing the activity of dsRNA to develop novel drugs for treatment various diseases, such as cancer, neuro- logical disorders, age-related macular degeneration viral infections. Small interfering (siRNA)-based have distinct advantages over conventional small molecule or protein-based drugs, including high specificity, higher potency reduced toxicity. However, there are several technical obstacles overcome before siRNA-based reach clinic. Delivery siRNA target tissues stability serum remain major challenge main focus current research development efforts. This review focused primarily on progress made developing RNAi therapeutics cancer challenges associ- ated with its clinical development. Use ligands recognizing cell-specific receptors achieve tumor-specific delivery siRNA, methods enhanced delivery, improving bioavailability pharmacokinetic properties reducing off-target effects non-specific discussed light evidence. Contents

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